Original articleThe chaperone activity and toxicity of Ambroxol (cas 18683-91-5) on Gaucher cells and normal mice

Add time:09/29/2019    Source:sciencedirect.com

Gaucher disease (GD), caused by a defect of acid β-glucosidase (β-Glu), is one of the most common sphingolipidoses. Recently, Ambroxol (cas 18683-91-5), an FDA-approved drug used to treat airway mucus hypersecretion and hyaline membrane disease in newborns, was identified as a chemical chaperone for GD. In the present study, we investigated the chaperone activity and toxicity of ambroxol on both cultured GD patient cells and normal mice. We found that ambroxol treatment significantly increased N370S, F213I, N188S/G193W and R120W mutant β-Glu activities in GD fibroblasts with low cytotoxicity. Additionally, we measured the β-Glu activity in the tissues of normal mice which received water containing increasing concentrations of ambroxol ad libitum for one week. No serious adverse effect was observed during this experiment. Ambroxol significantly increased the β-Glu activity in the spleen, heart and cerebellum of the mice. This result showed its oral availability and wide distribution and chaperone activity in the tissues, including the brain, and its lack of acute toxicity. These characteristics of ambroxol would make it a potential therapeutic chaperone in the treatment of GD with neurological manifestations.

We also recommend Trading Suppliers and Manufacturers of Ambroxol (cas 18683-91-5). Pls Click Website Link as below: cas 18683-91-5 suppliers

Prev:Original ArticleCiprofloxacin plus erythromycin or Ambroxol (cas 18683-91-5) ameliorates endotracheal tube-associated Pseudomonas aeruginosa biofilms in a rat model
Next:Ambroxol (cas 18683-91-5) as a pharmacological chaperone for mutant glucocerebrosidase)